Efficacy and Safety Study of RAD001 in the Growth of the Vestibular Schwannoma(s) in Neurofibromatosis 2 (NF2) Patients (AFINF2)
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Single Arm, Single Center, Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma|
- effect of RAD001 on the VS growth by MRI [ Time Frame: 1 year ] [ Designated as safety issue: No ]To determine whether RAD001 has an effect on the VS growth in patients with NF2 at a rate sufficient to submit the drug for further testing
- Effect of RAD001 on the volume of other intracranial tumors (MRI) and on hearing function (audiogram) [ Time Frame: 1, 2 and 4 years after inclusion in the study ] [ Designated as safety issue: Yes ]To determine if RAD001 has an effect on the volume of other intracranial tumors, to assess efficacy of RAD001 on hearing function, to determine whether RAD001 modulates signaling pathways in tumors removed during the course of the study and to determine long term safety
|Study Start Date:||January 2012|
|Estimated Study Completion Date:||July 2016|
|Estimated Primary Completion Date:||March 2016 (Final data collection date for primary outcome measure)|
Patient with Neurofibromatosis Type 2 and Vestibular Schwannoma treated with RAD001.
10 mg per os / day or 05mg per os / day with 12 month
Other Name: Afinitor / rapamycin
This protocol is a Phase II, open-label, efficacy and safety study of single-agent RAD001 in patients with NF2. During the study, subjects will receive continuous daily oral treatment with RAD001 for up to 4 years or until tumor progression.
Primary Objective: To determine whether RAD001 has an effect on the VS growth in patients with NF2 at a rate sufficient to submit the drug for further testing.
Secondary Objectives: To determine whether RAD001 has an effect on the volume of other intracranial tumors; to assess the effect of RAD001 on hearing function in patients with NF2 (when applicable); to determine whether RAD001 modulates signaling pathways in intracranial NF2 tumors removed during the course of the study. And determine the long term safety.
All patients will be treated with RAD001 10 mg p.o. daily dose (5mg if 15-17 years old with cutaneous surface area <1.5m2) for one year except in case of unacceptable toxicity, death, or discontinuation from the study for any other reason.
At 12 months an extension for another one year of RAD001 treatment will be discussed in case of response.
If the patient is stable (decrease or increase lower than 20%), treatment should be stopped and the patient should be kept under quarterly continuous surveillance. Resumption of treatment will be discussed case by case basis if tumor regrowth over 20% relative to the tumor volume at the end of treatment.
All patients will have a follow-up visit (including MRI) scheduled at 24 months after enrollment.
We modify the protocol to extend the treatment by RAD001 for a period of two additional years for stable patients still on treatment two years after enrollment.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01490476
|Hôpital Beaujon, 100 boulevard du Général Leclerc|
|Clichy, France, 92110|
|Principal Investigator:||Michel Kalamarides, Professor||Assistance Publique - Hôpitaux de Paris|